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557 articles
October 202624
Local ancestry-aware genome-wide meta-analysis uncovers novel genetic loci for sickle cell disease nephropathy.
This local ancestry-aware genome-wide association meta-analysis examined estimated glomerular filtration rate in participants from two well-characterized adult sickle cell disease cohorts, seeking genetic loci…
Denecimig (Mim8) prophylaxis once every 2 weeks for hemophilia A with or without inhibitors: 26-week results from FRONTIER4.
This open-label extension study evaluated every-2-week (Q2W) denecimig prophylaxis in patients aged ≥12 years with hemophilia A, with or without inhibitors, who had previously received denecimig in a phase 2 study and…
Crystalline κ light-chain proximal tubulopathy and Auer rod-like inclusions presenting as Fanconi syndrome: A case report.
A 53-year-old man presented with Fanconi syndrome, with hypokalemia, hypophosphatemia, mild metabolic acidosis, and renal glucosuria.
Variability in belumosudil exposure due to coadministration of voriconazole and isavuconazole in a patient with chronic graft-versus-host disease: Case report.
A man in his 50s with steroid-refractory pulmonary chronic graft-versus-host disease after cord blood transplantation received belumosudil, increased from 200 mg once daily to 200 mg twice daily.
Lymphoplasmacytic Lymphoma Involving Marrow-Containing Submandibular Heterotopic Ossification: A Case Report and Literature Review.
A 77-year-old man presented with a gradually enlarging, increasingly uncomfortable calcified nodule in the left submandibular region.
Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report.
A previously healthy 24-year-old man presented with dizziness, fatigue, and pancytopenia. Bone marrow examination supported aplastic anemia, with a PNH clone also identified.
Prognostic value of measuring LSC frequency in AML: a systematic review and meta-analysis.
This systematic review and reconstructed individual patient data meta-analysis assessed whether leukemia stem cell (LSC) measurements predict outcomes in patients with acute myeloid leukemia at diagnosis or in…
Ponatinib-Blinatumomab or Imatinib-Chemotherapy for Adult Ph-Positive ALL.
This multicenter, phase 3 randomized trial compared frontline ponatinib plus blinatumomab with imatinib plus chemotherapy in adults with Philadelphia-positive acute lymphoblastic leukemia.
Bacterial infections and antibiotic susceptibility in children with sickle-cell anemia on malaria chemoprevention using Dihydroartemisinin-piperaquine (DP) or Sulfadoxine-pyrimethamine (SP) in Uganda.
This analysis nested within a randomized, double-blind trial assessed bacterial infections and antimicrobial resistance among Ugandan children aged 6 months to 15 years with sickle-cell anemia receiving weekly…
TRANSFORM-1 phase 3 study: efficacy and safety of navitoclax plus ruxolitinib in patients with untreated myelofibrosis.
TRANSFORM-1 was a multicentre, randomised phase 3 trial comparing navitoclax plus ruxolitinib with placebo plus ruxolitinib in Janus kinase inhibitor-naïve adults with intermediate-2 or high-risk myelofibrosis.
A phase 2 trial of daratumumab monotherapy for patients with newly diagnosed cardiac stage IIIb AL amyloidosis.
This prospective, multicentre phase 2 trial evaluated daratumumab monotherapy in previously untreated patients with measurable AL amyloidosis and advanced cardiac stage IIIB disease; patients with inadequate response…
Aberrant splicing caused by a homozygous AK2 c.498 + 1G > A variant in a patient with reticular dysgenesis and concurrent B-cell acute lymphoblastic leukemia.
A boy with reticular dysgenesis (RD), a severe immunodeficiency associated with biallelic AK2 loss of function, was also diagnosed with B-cell acute lymphoblastic leukemia (B-ALL).
Association between elevated lipoprotein (a) and venous thromboembolism: A systematic review and meta-analysis.
This systematic review and meta-analysis examined observational evidence on elevated lipoprotein(a) [Lp(a)] and venous thromboembolism (VTE).
Myeloid neoplasm with PDGFRA rearrangement presenting in blast-phase with basophilia.
An adult man from Southern Africa presented with progressive fatigue. Blood morphology showed marked basophilia and 21% myeloblasts, with flow cytometry supporting a provisional diagnosis of acute myeloid leukaemia with…
Systematic review of the impact of germline mutations from unrelated bone marrow donors on post-allogeneic transplantation.
This systematic review examined whether germline mutations in unrelated bone marrow donors are associated with outcomes after allogeneic hematopoietic stem cell transplantation.
Hodgkin Lymphoma With Sarcoid-Like Reaction: Importance of Clinicopathological Correlation in Granulomatous Lymphadenopathy.
A 63-year-old man with systemic lymphadenopathy had a cervical lymph node biopsy showing epithelioid granulomas but no identifiable neoplastic cells, prompting an initial suspicion of sarcoidosis.
A case of immunofluorescence-negative light chain deposition disease diagnosed by mass spectrometry.
A 53-year-old man with kidney dysfunction and edema had nodular glomerular lesions on biopsy, but immunofluorescence did not show immunoglobulin or light-chain deposits.
The role of fibrinogen concentrate in the reduction of allogeneic blood product requirements: a systematic review and meta-analysis.
This systematic review and meta-analysis compared fibrinogen concentrate (FC) with other treatments for acquired hypofibrinogenaemia across clinical settings.
Unilateral adrenal haemorrhage during bridging anticoagulation in primary antiphospholipid syndrome: a case-based review.
A 42-year-old man with primary antiphospholipid syndrome (APS) and recurrent pulmonary embolism and deep vein thrombosis developed acute right upper-quadrant pain while receiving peri-procedural bridging anticoagulation…
Secondary pulmonary alveolar proteinosis mimicking progression of interstitial lung disease during treatment for adult-onset still's disease: An autopsy case report.
A 68-year-old woman with adult-onset Still’s disease-associated interstitial lung disease developed worsening respiratory failure while receiving immunosuppressive therapy, despite apparent control of systemic…
SF3B1-mutated MDS coexisting with β-thalassemia trait: A case report and literature review focused on diagnostic distinction from acquired β-thalassemia.
An 81-year-old man presented with fatigue and fever, and testing showed microcytic anemia. Bone marrow morphology and molecular studies identified SF3B1-mutated myelodysplastic syndrome (MDS) alongside β-thalassemia…
Increased mean corpuscular hemoglobin concentration: A case report as an early clue of chronic severe hyponatremia.
An 81-year-old woman was admitted after three days of altered mental status. Her complete blood count showed a markedly elevated mean corpuscular hemoglobin concentration (MCHC) of 369 g/L; the blood smear showed…
Relapsed/refractory PBML complicated with failed platelet implantation and mixed infection after allo-HSCT: A case report.
A 48-year-old woman had refractory non-germinal-center diffuse large B-cell lymphoma arising in the bone marrow, with p53 overexpression, a complex karyotype, and hemophagocytic lymphohistiocytosis.
Anticoagulation with the mechanistically distinct FXI/FXIa antibodies amrecibart (REGN9933A2) and cenvacibart (REGN7508Cat).
This Phase I randomised controlled trial programme evaluated two mechanistically distinct factor XI/FXIa monoclonal antibodies as potential anticoagulants, using in vitro assays, nonhuman primates and healthy…
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